Sapropterin Stada 100 mg soluble tablets EFG

Patient Information Leaflet

Introduction

Patient Information Leaflet

Sapropterine STADA 100 mg dispersible tablets EFG

sapropterin dihydrochloride

Read the entire leaflet carefully before you start taking this medicine, as it contains important information for you.

• Keep this leaflet, as you may need to read it again.
• If you have any questions, consult your doctor or pharmacist.
• This medicine has been prescribed for you only, and you should not give it to other people, even if they have the same symptoms as you, because it may harm them.
• If you experience any adverse effects, consult your doctor or pharmacist, even if they are adverse effects not listed in this leaflet. See section 4.

Package leaflet

1. What Sapropterin Stada is and what it is used for
2. What you need to know before taking Sapropterin Stada
3. How to take Sapropterin Stada
4. Possible side effects

5 Storage of Sapropterin Stada

1. Contents of the pack and other information

1. What Sapropterina Stada is and what it is used for

Sapropterina Stada contains the active substance sapropterin, which is a synthetic copy of a naturally occurring body substance called tetrahydrobiopterin (BH4). BH4 is required in the body to convert an amino acid called phenylalanine into another amino acid called tyrosine.

Sapropterin is used to treat hyperphenylalaninaemia (HPA) or phenylketonuria (PKU) in patients of any age. HPA and PKU are caused by abnormally high levels of phenylalanine in the blood, which can be harmful. Sapropterin reduces these levels in some patients who respond to BH4 and may help increase the amount of phenylalanine that can be included in the diet.

This medicine is also used to treat an inherited disorder called BH4 deficiency in patients of any age, in which the body cannot produce sufficient BH4.

Because BH4 levels are very low, the body cannot properly utilize phenylalanine, leading to increased levels of this amino acid, which may have harmful effects. By replacing the BH4 that the body cannot produce, sapropterin reduces the harmful excess of phenylalanine in the blood and increases dietary phenylalanine tolerance.

2. What you need to know before starting to take Sapropterin Stada

Do not take Sapropterin Stada

If you are allergic to sapropterin or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Consult your doctor or pharmacist before starting to take sapropterin, especially:

• if you are 65 years of age or older
• if you have kidney or liver problems
• if you are unwell. It is recommended to consult your doctor during illness, as blood phenylalanine levels may increase
• if you are predisposed to seizures

While receiving treatment with sapropterin, your doctor will perform blood tests to monitor your levels of phenylalanine and tyrosine, and to determine whether adjustments to the dose of sapropterin or your diet are necessary.

You must continue your dietary treatment according to your doctor's recommendations. Do not change your diet without informing your doctor. Even when taking sapropterin, if your blood phenylalanine concentrations are not well controlled, you may experience serious neurological problems. Your doctor must continue to monitor your blood phenylalanine concentrations regularly during treatment with sapropterin, to ensure that they are neither too high nor too low.

Use of Sapropterin Stada with other medicines

Inform your doctor or pharmacist if you are taking, have recently taken, or might need to take any other medicines. In particular, you must inform your doctor if you are using:

• levodopa (for the treatment of Parkinson's disease)
• medicines for the treatment of cancer (e.g., methotrexate)
• medicines for the treatment of bacterial infections (e.g., trimethoprim)
• medicines that cause blood vessel dilation (e.g., glyceryl trinitrate (GTN), isosorbide dinitrate (ISDN), sodium nitroprusside (SNP), molsidomine, minoxidil).

Pregnancy and breastfeeding

If you are pregnant or breastfeeding, think you may be pregnant, or are planning to become pregnant, consult your doctor or pharmacist before using this medicine.

If you are pregnant, your doctor will advise you on how to properly control your phenylalanine levels. If these levels are not strictly controlled before pregnancy or when you become pregnant, it may be harmful to you and your baby. Your doctor will monitor the restriction of phenylalanine intake through diet before and during pregnancy.

If a strict diet does not satisfactorily reduce the amount of phenylalanine in the blood, your doctor will consider whether you should take this medicine.

You must not use this medicine during breastfeeding.

Driving and operating machinery

Sapropterin is not expected to affect the ability to drive and operate machinery.

Important information about some of the components of Sapropterin Stada

This medicine contains less than 1 mmol of sodium (23 mg) per tablet; that is, essentially "sodium-free".

3. How to take Sapropterin Stada

Follow exactly the instructions for use of this medicine given by your doctor. If in doubt, consult your doctor again.

Dosage for PKU

The recommended starting dose of sapropterin in patients with PKU is 10 mg per kg of body weight. Take sapropterin once daily with a meal to enhance absorption, at the same time each day, preferably in the morning. Your doctor may adjust the dose, usually between 5 and 20 mg per kg daily, depending on your condition.

Dosage for BH4 Deficiency

The recommended initial dose of sapropterin in patients with BH4 deficiency is 2 to 5 mg per kg of body weight. Take sapropterin with a meal to increase absorption. Divide the total daily dose into 2 or 3 doses administered throughout the day. Your doctor may adjust the dose up to 20 mg per kg per day, depending on your condition.

The following table is an example of how to calculate the appropriate dose

Administration Method

For patients with PKU, the total daily dose should be taken once daily at the same time each day, preferably in the morning.

For patients with BH4 deficiency, the total daily dose should be divided into 2 or 3 doses taken throughout the day.

Use in all patients

Place the prescribed tablets into a glass or cup of water, exactly as described below, and stir until dissolved.

The tablets may take a few minutes to dissolve. To speed up dissolution, you may crush the tablets. Small particles may be visible in the solution; these will not affect the effectiveness of the medicine. Drink the dissolved sapropterin preparation with a meal within 15 to 20 minutes after preparation.

Do not ingest the desiccant capsule included in the bottle.

Use in patients with body weight above 20 kg

Place the tablets into a glass or cup (120 to 240 ml) of water and stir until dissolved.

Use in children with body weight up to 20 kg

The dose is based on body weight, which will change as the child grows. Your doctor will advise you on:

• the number of sapropterin tablets required for one dose
• the amount of water needed to mix one dose of sapropterin
• the volume of solution to administer to the child for the prescribed dose

The child should drink the sapropterin solution with a meal.

Administer the prescribed volume of solution to the child within 15 to 20 minutes after dissolving the tablets. If the dose cannot be administered to the child within 15 to 20 minutes after tablet dissolution, a new solution must be prepared, as unused solution must not be used after 20 minutes.

Materials required to prepare and administer the sapropterin dose to the child

• The number of sapropterin tablets required for one dose
• A dosing cup with graduations marked at 20, 40, 60, and 80 ml
• A glass or cup
• A clean spoon or stirring utensil
• An oral dosing syringe (graduated in 1 ml increments) (a 10 ml oral syringe for administering volumes ≤10 ml or a 20 ml oral syringe for administering volumes >10 ml)

Ask your doctor for the dosing cup to dissolve the tablets and the 10 ml or 20 ml oral syringe if you do not have these materials.

Steps for preparing and administering the dose:

• Place the prescribed tablets into the dosing cup. Add the amount of water specified by your doctor (e.g., your doctor instructed you to use 20 ml to dissolve one sapropterin tablet). Ensure the liquid level aligns with the corresponding graduation mark on the dosing cup as directed by your doctor. Stir with a clean spoon or utensil until the tablets are dissolved.
• If your doctor instructed you to administer only part of the solution, insert the tip of the oral dosing syringe into the dosing cup. Slowly pull back the plunger to draw up the volume specified by your doctor.
• Then transfer the solution to a glass or administration cup by slowly pushing the plunger until all the solution previously drawn into the oral dosing syringe is dispensed (e.g., if your doctor instructed you to dissolve two sapropterin tablets in 40 ml of water and administer 30 ml to the child, you will need to use the 20 ml oral dosing syringe twice to withdraw and transfer the 30 ml of solution (e.g., 20 ml + 10 ml) into a glass or cup). Use a 10 ml oral syringe for volumes ≤10 ml or a 20 ml oral syringe for volumes >10 ml.
• If the infant is too young to drink from a glass or cup, you may administer the solution using the oral dosing syringe. Draw up the prescribed volume of prepared solution from the dosing cup and insert the tip of the oral dosing syringe into the infant’s mouth. Aim the tip of the syringe toward one of the cheeks. Slowly push the plunger so that a small amount is released at a time, until all the solution in the oral dosing syringe has been administered.
• Discard any remaining unused solution. Remove the plunger from the body of the oral dosing syringe. Wash both parts of the oral syringe and the dosing cup with warm water and allow them to air dry. Once the oral syringe is dry, reinsert the plunger into the syringe barrel. Store the oral syringe and dosing cup for the next use.

If you take more Sapropterina Stada than you should

If you take more sapropterin than prescribed, you may experience adverse effects, which may include headache and dizziness. If you take more sapropterin than prescribed, inform your doctor or pharmacist immediately.

In case of overdose or accidental ingestion, contact your doctor or pharmacist immediately or call the Toxicology Information Service at telephone number: 91 562 04 20, indicating the medication and the amount ingested.

If you forget to take Sapropterin Stada

Do not take a double dose to make up for missed doses. Take the next dose at your usual time.

If you interrupt treatment with Sapropterin Stada

Do not stop treatment with sapropterin without first discussing it with your doctor, as blood phenylalanine levels may increase.

If you have any further questions about the use of this medicine, ask your doctor or pharmacist.

4. Possible adverse effects

Like all medicines, this medicine can cause adverse effects, although not everybody gets them.

A few cases of allergic reactions (such as skin rash and serious reactions) have been reported. Their frequency is unknown (frequency cannot be estimated from the available data).

If you experience inflamed, red and intensely itchy areas (urticaria), runny nose, rapid or irregular pulse, swelling of the tongue or throat, sneezing, wheezing, severe breathing difficulty, or dizziness, you may be experiencing a serious allergic reaction to this medicine. If you have these symptoms, consult your doctor immediately.

Very common adverse effects (may affect more than 1 in 10 people):

Headache and runny nose.

Common adverse effects (may affect up to 1 in 10 people):

Sore throat, nasal congestion or stuffiness, cough, diarrhoea, vomiting, stomach pain, blood phenylalanine levels too low, indigestion, and general discomfort (nausea) (see section 2: "Warnings and precautions").

Adverse effects frequency not known (cannot be estimated from the available data):

Gastritis (inflammation of the stomach lining), oesophagitis (inflammation of the oesophageal lining).

Reporting of adverse reactions

If you experience any type of adverse reaction, consult your doctor, pharmacist, or nurse, even if it is a possible adverse reaction not listed in this leaflet. You may also report them directly through the Spanish Pharmacovigilance System for Human Medicinal Products: www.notificaRAM.es. By reporting adverse reactions, you can help provide more information on the safety of this medicine.

5. Storage of Sapropterin Stada

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date stated on the bottle and the carton, following EXP. The expiry date refers to the last day of the month indicated.

HDPE bottles with desiccant and polyester coil, closed with PP cap:

Keep the bottle tightly closed and store in the original packaging to protect it from moisture.

Al/PVC/Al/OPA blisters, Al/PVC/Al/OPA unit-dose blisters:

This medicine does not require any special storage conditions.

Medicines must not be disposed of via wastewater or household waste. Dispose of unused medicines and their containers at the SIGRE Point in your pharmacy. If you have any doubts, ask your pharmacist how to properly dispose of medicines and their packaging. This will help protect the environment.

6. Contents of the container and additional information

• The active substance is sapropterin dihydrochloride. Each tablet contains 100 mg of sapropterin dihydrochloride (equivalent to 77 mg of sapropterin).
• The other components are mannitol (E421), crospovidone, calcium hydrogen phosphate, ascorbic acid (E300), riboflavin (E101), and stearoyl fumarate sodium.

Appearance of the medicinal product and contents of the pack

Sapropterin Stada 100 mg dispersible tablets EFG are 10 mm round, white to slightly yellowish tablets, with an embossed "C" on one side.

Sapropterin Stada 100 mg dispersible tablets EFG are available in:

• HDPE bottles with desiccant and polyester coil sealed with PP cap.

Pack sizes: 30 and 120 tablets

• Al/PVC/Al/OPA blisters

Pack sizes: 30 and 120 tablets

• Monodose Al/PVC/Al/OPA blisters

Pack sizes: 30x1 and 120x1 unit dose tablets

Only certain pack sizes may be marketed.

Marketing Authorization Holder and Manufacturing Responsible

Marketing Authorization Holder

Laboratorio STADA, S.L.

Frederic Mompou, 5

08960 Sant Just Desvern (Barcelona)

Spain

[email protected]

Manufacturer Responsible

STADA Arzneimittel AG,

Stadastrasse 2 - 18,

61118 Bad Vilbel,

Germany

or

Coripharma ehf.,

Reykjavikurvegur 78,

IS -220 Hafnarfjordur,

Iceland

or

Centrafarm Services B.V,

Van de Reijtstraat 31 E,

4814NE Breda,

Netherlands

This medicinal product is authorized in the Member States of the European Economic Area under the following names:

Denmark Sapropterin STADA

Germany Sapropterin AL 100 mg Tablets for the preparation of an oral solution

Iceland Sapropterin STADA 100 mg dispersible tablets

Netherlands Sapropterin diHCl CF 100 mg, dispersible tablets

Norway Sapropterin STADA 100 mg dispersible tablets

Spain Sapropterin STADA 100 mg dispersible tablets EFG

Sweden Sapropterin STADA 100 mg dispersible tablets

Date of latest review of this leaflet: December 2023

Detailed information about this medication is available on the website of the Spanish Agency of Medicines and Medical Devices (AEMPS): http://www.aemps.gob.es/